William W. Hauswirth

Gene therapy restores vision in a canine model of childhood blindness

Gregory M. Acland, Jharna Ray, Qí Zhāng et al. · Nature Genetics · 2001 · 1.2K citations

Medicine, Ophthalmology, Vision Science +16

Treatment of Leber Congenital Amaurosis Due to <i>RPE65</i> Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

William W. Hauswirth, Tomás S. Alemán, Shalesh Kaushal et al. · Human Gene Therapy · 2008 · 947 citations · Full text

Medicine, Molecular Biology, Ophthalmology +15

A Muscleblind Knockout Model for Myotonic Dystrophy

Rahul Kanadia, Karen A. Johnstone, Ami Mankodi et al. · Science · 2003 · 748 citations

Life Sciences, Genetics, Molecular Genetics +16

Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

Artur V. Cideciyan, Tomás S. Alemán, Sanford L. Boye et al. · Proceedings of the National Academy of Sciences · 2008 · 664 citations · Full text

Medicine, Genetics, Ophthalmology +17

A "humanized" green fluorescent protein cDNA adapted for high-level expression in mammalian cells

Sergei Zolotukhin, Mark Potter, William W. Hauswirth et al. · Journal of Virology · 1996 · 614 citations · Full text

Pharmacy, Biotechnology, Molecular Biology +17